Clinical Trial: Pilot Trial of Deferasirox in the Treatment of Porphyria Cutanea Tarda
Study Status: Completed
Recruit Status: Completed
Study Type: Interventional
Official Title: Pilot Trial of Deferasirox in the Treatment of Porphyria Cutanea Tarda
Brief Summary:
To determine the efficacy and tolerability of deferasirox in the treatment of Porphyria Cutanea Tarda.
Primary objective - the elimination of all blistering within 6 months of treatment.
Secondary objective - decrease in total body iron levels.
Detailed Summary:
Phlebotomy is the standard therapy for Porphyria Cutanea Tarda (PCT), but it can be inconvenient and cause anemia in some patients.
Deferasirox is a new class of tridentate iron chelators with high affinity and selectivity for iron. The medication is administered orally, which if effective for PCT would make it a more convenient and possibly more tolerable option for patients.
Sponsor: University of Texas Southwestern Medical Center
Current Primary Outcome: Number of Participants Showing Reduction or Elimination of Skin Blistering [ Time Frame: Within 6 months of treatment. ]
Original Primary Outcome: The elimination of all skin blistering. [ Time Frame: Within 6 months of treatment. ]
Current Secondary Outcome: Number of Participants Showing Decrease in Ferritin and Urinary Porphyrin Level [ Time Frame: 6 months ]
Original Secondary Outcome: Assess for a decrease in total body iron levels along with efficacy, safety and tolerability of deferasirox. [ Time Frame: 6 months ]
Information By: University of Texas Southwestern Medical Center
Dates:
Date Received: January 10, 2008
Date Started: January 2008
Date Completion:
Last Updated: January 9, 2014
Last Verified: January 2014